亨廷顿病的基因治疗。

Gene therapy for Huntington's disease.

机构信息

Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.

出版信息

Neurobiol Dis. 2012 Nov;48(2):243-54. doi: 10.1016/j.nbd.2011.12.030. Epub 2011 Dec 24.

DOI:10.1016/j.nbd.2011.12.030

Abstract

Huntington's disease (HD) is a neurodegenerative disease for which there is no cure. Therapies that are efficacious in animal models have to date shown benefit for humans. One potential powerful approach is gene therapy. The ideal method of administration of gene therapy has been hotly debated and viral vectors have provided one method of long-term and wide-spread delivery to the brain. Trophic factors to protect cells from degeneration and RNAi to reduce mutant huntingtin (mHtt) protein expression are 2 main classes of compounds that demonstrate benefit in animal models. This review will examine some commonly used adeno-associated viral (AAV) vectors and discuss some therapies that hold promise for HD.

摘要

亨廷顿病（HD）是一种神经退行性疾病，目前尚无治愈方法。在动物模型中有效的治疗方法迄今为止已显示对人类有益。一种潜在的有效方法是基因治疗。基因治疗的理想给药方法一直存在激烈争论，病毒载体为向大脑提供长期和广泛的递药提供了一种方法。营养因子可保护细胞免受变性，RNAi 可减少突变亨廷顿蛋白（mHtt）的表达，这是在动物模型中显示出益处的 2 种主要化合物。本综述将检查一些常用的腺相关病毒（AAV）载体，并讨论一些对 HD 有希望的治疗方法。